Investigational Trial to Evaluate XC001 Delivered Via an Cardiac Catheter in Subjects With Chronic Angina.

Study Summary

This is a two-part study, comprised of an initial open-label run-in phase (Part 1) in a subset of 3 subjects to provide first data regarding safety, and feasibility of the percutaneous endovascular catheter-facilitated intramyocardial delivery of XC001 in patients with RA due to obstructive CAD. Part 1 of the study is comprised of 3 subjects with RA (CCS class II-IV) who will receive 4×1011 viral particles (vp) XC001. An Independent Data Monitoring Committee (IDMC), the committee will review safety and feasibility data and approval to commence enrollment in Part 2 of the study. Part 2 is a randomized, double-blind, sham-procedure control study. Subjects with RA (CCS class II-IV) with no therapeutic options will be randomized 1:1 to either the treatment group with catheter delivery of 4×1011 vp XC001 (approximately N=53) or a sham procedure group (approximately N=53). It is estimated that approximately 106 subjects will be randomized to result in 100 evaluable subjects. All subjects enrolled in Part 1, as well as Part 2 will follow all screening and safety monitoring procedures for up to 12 months (Table 2), and will be included in the safety analysis of the study.

Primary Outcome

The primary objective of this study is to evaluate the effect of XC001 on a composite endpoint of a) TED from a graded treadmill test (ETT), as assessed by a core lab, b) angina frequency from a diary (extracting the 2 weeks prior to the Week 12 and Week 26 visit) and c) ischemic burden, as quantified by stress imaging by PET (as assessed by a core lab) at 12 and 26 weeks following a one-time endocardial administration to CAD patients with RA compared to the sham procedure.

Conditions Studied

Interventions

• COMBINATION_PRODUCT XC001
• COMBINATION_PRODUCT Sham (No Treatment)

Study Locations (1)